Quick Facts Field Details Active Ingredient Patisiran Drug Category Rare / Orphan Diseases Primary Use Hereditary transthyretin amyloidosis (siRNA gene silencer) Availability Available in Turkey ✓ Introduction Patisiran is a...
Quick Facts Field Details Active Ingredient Inotersen Drug Category Rare / Orphan Diseases Primary Use Hereditary transthyretin amyloidosis (antisense oligonucleotide) Availability Available in Turkey ✓ Introduction Inotersen is a Rare...
Quick Facts Field Details Active Ingredient Tafamidis Drug Category Rare / Orphan Diseases Primary Use Transthyretin amyloid cardiomyopathy (TTR stabilizer) Availability Available in Turkey ✓ Introduction Tafamidis is a Rare...
Quick Facts Field Details Active Ingredient Edaravone Drug Category Rare / Orphan Diseases Primary Use ALS/motor neuron disease (neuroprotective, free radical scavenger) Availability Available in Turkey ✓ Introduction Edaravone is...
Quick Facts Field Details Active Ingredient Riluzole (ALS) Drug Category Rare / Orphan Diseases Primary Use ALS (glutamate antagonist, slows disease progression) Availability Available in Turkey ✓ Introduction Riluzole (ALS)...
Quick Facts Field Details Active Ingredient Deflazacort Drug Category Rare / Orphan Diseases Primary Use Duchenne muscular dystrophy (corticosteroid) Availability Available in Turkey ✓ Introduction Deflazacort is a Rare /...
Quick Facts Field Details Active Ingredient Ataluren Drug Category Rare / Orphan Diseases Primary Use Duchenne muscular dystrophy (nonsense mutation read-through) Availability Available in Turkey ✓ Introduction Ataluren is a...
Quick Facts Field Details Active Ingredient Elexacaftor/Tezacaftor/Ivacaftor Drug Category Rare / Orphan Diseases Primary Use Cystic fibrosis (triple CFTR modulator — most effective) Availability Available in Turkey ✓ Introduction Elexacaftor/Tezacaftor/Ivacaftor...
Quick Facts Field Details Active Ingredient Tezacaftor/Ivacaftor Drug Category Rare / Orphan Diseases Primary Use Cystic fibrosis (CFTR modulator combination) Availability Available in Turkey ✓ Introduction Tezacaftor/Ivacaftor is a Rare...
Quick Facts Field Details Active Ingredient Ivacaftor Drug Category Rare / Orphan Diseases Primary Use Cystic fibrosis (CFTR potentiator, specific mutations) Availability Available in Turkey ✓ Introduction Ivacaftor is a...